Rare Diseases
-
Plain language summary of a study looking at heart muscle thickness and kidney function in women with Fabry disease who received agalsidase beta treatment
The new plain language summary of publication in Future Cardiology uses information from the Fabry Registry to understand the effects of agalsidase beta treatment in women with Fabry disease.
-
Prednisone and deflazacort in Duchenne muscular dystrophy: a patient perspective and plain language summary publication of the Cincinnati study
This plain language summary of publication, co-authored by a patient, a caregiver and a treating physician, includes the patient perspective and help patients with Duchenne muscular dystrophy and their caregivers understand the results of the Cincinnati study.
-
Plain language summary of the MPOWERED trial comparing the effectiveness, safety, and patient experiences’ of monthly injections and oral medication used to treat acromegaly
The MPOWERED study looked oral octreotide as a treatment for patients with acromegaly. This plain language summary can help people understand the findings from this study.
-
Plain language summary of the long-term effect of ataluren in patients with Duchenne muscular dystrophy
Read the latest plain language summary looking at how ataluren treatment in combination with standard of care can delay important signs of disease progression in patients with Duchenne muscular dystrophy.
-
Vosoritide treatment accelerates bone growth in children with achondroplasia
Read the article summarising the results of two studies looking at vosoritide as a potential treatment for children with achondroplasia.
-
ENLIVEN study: Pexidartinib for tenosynovial giant cell tumor (TGCT)
The first of it’s kind published in a peer-reviewed journal, this article published in Future Oncology provides a lay language summary of the ENLIVEN study.